ZIB

1

Electronic Resource

Prevalence of inhibitor formation in a cohort of haemophilic children exposed to several products of various purities (1995)

ARONIS, S. ; PLATOKOUKI, H. ; KAPSIMALI, Z. ; [et al.]

Oxford, UK : Blackwell Publishing Ltd

Haemophilia 1 (1995), S. 0

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ISSN: 1365-2516

Source: Blackwell Publishing Journal Backfiles 1879-2005

Topics: Medicine

Notes: Summary. To evaluate the frequency and potency of inhibitor formation based on the product used, we retrospectively reviewed the records of 99 children with various types and severity of haemophilia (haemophilia A 82, severe 46; haemophilia B 10, severe 6; vWD 7) treated for the last 20 years. After a mean observation period of 8 years an overall of 23 patients (23.2′/0) developed an inhibitor (haernophilia A 26.8%; severe 4O%, moderate 20%, mild 3.8%). None of the haemophilia B patients presented with an inhibitor, and only one child with bevere vWD (1/7, 14.3%) showed a transient inhibitor under cryoprecipitate therapy. Inhibitor titre was low (〈 5 BU) in most cases (91.3%) and in only two patients (8.75%) was 6 and 8 BU respectively. Antibodies to FVIII were transient (detected only once) in four (17.4%) and intermittent in 19 patients (82.6%). By the age of 12 years, 17/23 patients (73.9%) had demonstrated an inhibitor. The inhibitor detection seemed to be higher in the groups of patients exposed to monoclonal (3115, 20%), SID-treated (10159, 16.9%) or H/T FVIII concentrates (6/41, 14.6%), compared to groups of patients who received cryo/plasma (9.5%) or unmodified concentrates (5.1%); nevertheless the differences were not statistically significant.Surprisingly, none of the 52 patients who received a S/D + chromatography-treated factor VIIl concentrate developed an inhibitor after a mean observation period of 1.7 years (range 0.2–2 years). The overall prevalence of inhibitor formation in previously untreated haemophiliacs was 14.3% (4/28), irrespective of the product used.Our data indicate that a high proportion of our haemophilic children exposed to several products of various purities have developed a low-titre inhibitor which in most cases was transient or intermittent. However, despite the presence of the antibody, none of the patients needed a change in the mode of treatment.

Type of Medium: Electronic Resource

URL: http://dx.doi.org/10.1111/j.1365-2516.1995.tb00082.x

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2

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Pharmacokinetics of activated recombinant coagulation factor VII (NovoSeven®) in children vs. adults with haemophilia A (2004)

Villar, A. ; Aronis, S. ; Morfini, M. ; [et al.]

Oxford, UK : Blackwell Science Ltd

Haemophilia 10 (2004), S. 0

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ISSN: 1365-2516

Source: Blackwell Publishing Journal Backfiles 1879-2005

Topics: Medicine

Notes: Summary. To establish the pharmacokinetic profile of activated recombinant coagulation factor VII (rFVIIa; NovoSeven®) in children with haemophilia A, and to compare it with the pharmacokinetic profile in adults with haemophilia A.Twelve children (2–12 years) received one single dose of rFVIIa 90 and 180 μg kg−1 in randomized order separated by a washout period of 48 h to 1 month. Six adults (18–55 years) received a single dose of rFVIIa 90 μg kg−1. The pharmacokinetic analyses were based on a non-compartmental method.In children, the plasma level of FVII increased proportionally with the dose. The total body clearance normalized for body weight was significantly faster in children than in adults (FVII:C, 58 vs. 39 mL kg−1 h−1 and FVIIa, 78 vs. 53 mL kg−1 h−1, P 〈 0.05). A trend towards a larger volume of distribution at steady-state in children than in adults was observed (P 〉 0.05).Dose proportionality was established for plasma concentrations of FVII in children with haemophilia A at the dose levels investigated (90 and 180 μg kg−1 rFVIIa). Following administration of rFVIIa 90 μg kg−1, significantly faster clearance was observed in children compared with adults, suggesting that higher doses of rFVIIa may be needed to achieve the same plasma levels as in adults.

Type of Medium: Electronic Resource

URL: http://dx.doi.org/10.1111/j.1365-2516.2004.00925.x

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3

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Characterization of mutations in the factor VIII gene by direct sequencing of amplified genomic DNA

Higuchi, M. ; Wong, C. ; Kochhan, L. ; [et al.]

Amsterdam : Elsevier

Genomics 6 (1990), S. 65-71

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ISSN: 0888-7543

Source: Elsevier Journal Backfiles on ScienceDirect 1907 - 2002

Topics: Biology , Medicine

Type of Medium: Electronic Resource

URL: http://dx.doi.org/10.1016/0888-7543(90)90448-4

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4

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Mild hemophilia A associated with a cryptic donor splice site mutation in intron 4 of the factor VIII gene

Youssoufian, H. ; Kazazian, H.H. ; Patel, A. ; [et al.]

Amsterdam : Elsevier

Genomics 2 (1988), S. 32-36

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ISSN: 0888-7543

Source: Elsevier Journal Backfiles on ScienceDirect 1907 - 2002

Topics: Biology , Medicine

Type of Medium: Electronic Resource

URL: http://dx.doi.org/10.1016/0888-7543(88)90106-1

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5

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Haemostatic variables in phenylketonuric children under dietary treatment (1996)

Schulpis, K. H. ; Platokouki, H. ; Papakonstantinou, E. D. ; [et al.]

Springer

Journal of inherited metabolic disease 19 (1996), S. 603-609

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ISSN: 1573-2665

Source: Springer Online Journal Archives 1860-2000

Topics: Medicine

Notes: Summary Classical phenylketonuria (PKU) (McKusick 261600) is an inborn error of metabolism treated by a controlled low-phenylalanine (Phe) diet started as soon as possible in the first days of life. Such a diet can be achieved with vegetable protein and can be considered non-atherogenic because of the reduction of animal products. Thirty patients with PKU were classified into two groups according to their annual mean Phe levels. Their daily protein intake was largely replaced by PKU2 Milupa which contains a mixture of amino acids. The product has no phenylalanine or fat of any kind. Thirty-eight (38) individuals of comparable age were used as controls. Group A (n=15) had good compliance with the special diet (Phe mean 192±115 µmol/L); group B (n=15) did not strictly adhere to the diet (Phe mean 595±263 µmol/L). Certain haemostatic components (factors I, VII, VIII, and X, antithrombin III, protein C, and plasminogen) and lipid variables (cholesterol, triglycerides, high-density lipoprotein, low-density lipoprotein, very-low-density lipoprotein) as well as Phe levels were estimated. All the haemostatic factors studied were found within the normal range with the exception of a significant reduction in protein C in both groups of PKU patients. Furthermore, a statistically significant reduction in factor VII and X concentrations was observed in patients on strict diet. Cholesterol and low-density lipoprotein concentrations were significantly lower in PKU children compared to normal controls. It is suggested that even though the special diet of PKU children, especially in group A, is rich in vegetables, the reduced fat intake might have impaired the absorption of vitamin K and its delivery to the site of synthesis of vitamin K-dependent haemostatic factors.

Type of Medium: Electronic Resource

URL: http://dx.doi.org/10.1007/BF01799833

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